Hydroxyurea to prevent brain injury in children with sickle cell disease (HU Prevent)-A randomized, placebo-controlled phase II feasibility/pilot study.
Publication/Presentation Date
10-1-2024
Abstract
Central nervous system (CNS) injury is common in sickle cell disease (SCD) and occurs early in life. Hydroxyurea is safe and efficacious for treatment of SCD, but high-quality evidence from randomized trials to estimate its neuroprotective effect is scant. HU Prevent was a randomized (1:1), double-blind, phase II feasibility/pilot trial of dose-escalated hydroxyurea vs. placebo for the primary prevention of CNS injury in children with HbSS or HbS-β
Volume
99
Issue
10
First Page
1906
Last Page
1916
ISSN
1096-8652
Published In/Presented At
Casella, J. F., Furstenau, D. K., Adams, R. J., Brambilla, D. J., Lebensburger, J. D., Fehr, J. J., Jordan, L. C., King, A. A., Ichord, R. N., McKinstry, R. C., Kraut, M. A., Shaw, D. W., White, D. A., Whyte-Stewart, D. A., Avadhani, R., Barron-Casella, E. A., Cannon, A. D., Eaton, C. K., Riekert, K. A., Shay, J. E., … Hanley, D. F. (2024). Hydroxyurea to prevent brain injury in children with sickle cell disease (HU Prevent)-A randomized, placebo-controlled phase II feasibility/pilot study. American journal of hematology, 99(10), 1906–1916. https://doi.org/10.1002/ajh.27423
Disciplines
Medicine and Health Sciences | Pediatrics
PubMedID
38953438
Department(s)
Department of Pediatrics
Document Type
Article